2008 LYSOSOMAL DISEASES AND THE BRAIN CONFERENCE
a conference hosted by The Children's Gaucher Research Fund

2008 Conference Agenda (draft)

All Times are Pacific Standard Time
Thursday, May 29, 2008
12:00p.m. to 3:00p.m. Conference Registration
3:00 p.m. to 3:10p.m. Welcome, Greg Macres (CGRF)
Session 1. Basic science – pathological mechanisms. Chairs: Tony Futerman Ph.D. and Col. Kondi Wong M.D.
3:10p.m. to 3:30p.m. “Overview of basic science session” Tony Futerman Ph.D. (Weizmann Institute of Science – Rehovot – Israel)
3:30 p.m. to 4:15 p.m. “Molecular Mechanisms of Lysosome Biogenesis” Juan S. Bonifacino, Ph. D. - National Institutes of Health - Bethesda, Maryland
4:15. p.m to 5:00 p.m. “Fate and function of glucosylceramide in mammalian cells” Gerrit van Meer, Ph. D. - Utrecht University - The Netherlands
5:00 p.m. to 5:20 p.m “The role of defective calcium-homeostasis in the neuropathology of type 2 and 3 Gaucher disease”. Einat Vitner, Ms.c (Weizmann Institute of Science - Rehovot – Israel)
5:20p.m. to 5:35 p.m. TBD: Selected from Poster Board Abstracts
6:30p.m. to 9:00p.m. Cocktail Reception and Dinner - Poolside
Friday May 30, 2008
7:00a.m. to 8:00a.m. Continental Breakfast
8:00a.m. to 8:05a.m. Opening Remarks
Session 1. (Continued) Basic science – pathological mechanisms. Chairs: Tony Futerman Ph.D. and Col. Kondi Wong M.D.
8:05a.m. to 8:50a.m. “Glucosylcerebroside accumulation in gba2 knockout mouse” Yildiz Yildiz, M.D. - University of Bonn, Bonn - Germany
8:50a.m. to 9:35a.m. Novel metabolic pathway of glucosylceramide involving Klotho-related protein: Insights into Gaucher disease Makoto Ito, Ph. D. - Kyushu University - Japan
9.35a.m. to 9.50 TBD: Selected from Poster Board Abstracts
9:50a.m. to 10:20a.m. Coffee Break
10.20a.m. to 11.05a.m. “Autophagy in the pathophysiology of the brain” Ana Maria Cuervo M.D. Ph.D – Albert Einstein College of Medicine – Bronx – New York
11.05a.m. to 11.50a.m. “GM1-ganglioside as apoptotic signal in ER- and mitochondria-mediated cell death in neurodegenerative GM1 gangliosidosis” Alessandra d'Azzo , Ph.D. (St. Jude Children’s Research Hospital - Memphis, Tennessee
11.50a.m. to 12.05p.m. TBD: Selected from Poster Board Abstracts
12:05p.m. to 12:50p.m. Lunch
12:50p.m. to 1:35p.m. “Common themes in Batten disease pathogenesis” Jonathon Cooper, Ph. D. - King's College – London
1.35p.m. to 1.50p.m. TBD: Selected from Poster Board Abstracts
1:50p.m. to 2.30p.m. Basic Science – Panel Discussion With all Presenters – Q&A Moderators: Tony Futerman Ph.D. and Col. Kondi Wong M.D.
2:30p.m. to 3:15p.m. Gaucher disease and Parkinsonism Ellen Sidransky, M.D. – National Institutes of Health – Bethesda, Maryland
Moderator: Gregory A. Grabowski, M.D.
3:15p.m. to 5:15p.m. Poster Session
6:00p.m. to 9:00p.m. Cocktail Reception/Dinner
9:00p.m. Desert Wine Served on the Veranda
Saturday, May 31, 2008
7:30a.m. to 8:30a.m. Continental Breakfast
8:30a.m. to 8:45a.m. Opening Remarks and overview Gregory A. Grabowski, M.D.
8:45a.m. to 9:30a.m. “Therapeutic trials and pathogenetic studies in an animal model of metachromatic leukodystrophy” Volkmar Gieselmann M.D. - University of Bonn - Bonn, Germany
9:30a.m. to 10:15a.m. “New therapeutic approaches for lysosomal disorders of the brain” Fran Platt, Ph. D. - University of Oxford, United Kingdom
10:15a.m. to 10:45a.m. Coffee Break
10:45a.m. to 11:30a.m. “Prospects for Treating the CNS Disease in LSDs” Seng H. Cheng , Ph. D. - Genzyme Corporation - Framingham, Massachusetts
11:30a.m. to 12:15p.m. “Re-Engineering Lysosomal Enzymes for Intravenous Delivery to the Human Brain” William M. Pardridge, M.D. (Bill) - University of California at Los Angeles - Los Angeles, California
12.15p.m. to 12.30p.m TBD: Selected from Poster Board Abstracts
12:30p.m. to 1:15p.m. Lunch
1:15 p.m. to 2:00p.m. “Pharmacological chaperones as a potential therapy for lysosomal disorders of the brain” Brandon A. Wustman, Ph. D. - Amicus Therapeutics – Cranbury – NJ
2:00p.m. to 2:15p.m. TBD: Selected from Poster Board Abstracts
2:15p.m. to 3:00p.m. Therapy – Panel Discussion With all Presenters – Q&A Moderators: Roscoe Brady M.D. and Raphael Schiffmann M.D.

Contact Us: Tel: 916.797.3700 • Fax: 916.797.3707 • e-mail: research@childrensgaucher.org
P.O. Box 2123 • Granite Bay, California 95746-2123